Months after a clinical failure in Alzheimer's disease destroyed most of its value, Axovant Sciences Ltd. (NASDAQ:AXON) is seeking a path forward by obtaining rights to a gene therapy for Parkinson's disease.
Oxford BioMedica plc (LSE:OXB) granted Axovant exclusive, worldwide rights to OXB-102, its lentiviral gene therapy designed to stimulate dopamine production in the putamen. Axovant, which renamed the program AXO-Lenti-PD, intends to start a Phase I/II dose-escalation study of the therapy by YE18 in patients with advanced PD.
Axovant shares were up $2.45 (140%) to $4.20 in premarket trading Wednesday. The company's closing price Tuesday of $1.75 was 94% below its 52-week high of $27.28, which it hit days before the company's intepirdine (RVT-101) missed the co-primary endpoints in a Phase III trial to treat mild to moderate AD. The candidate later failed in two Phase II studies in dementia (see BioCentury Extra, Sept. 26, 2017 & Jan. 8, 2018).
In Wednesday's deal, Axovant will pay Oxford BioMedica $30 million up front, including $5 million as a credit for process development work and clinical supply of the therapy. Oxford BioMedica is eligible for $55 million in development milestones and $757.5 million in regulatory and sales milestones, plus tiered royalties up to 10%.
Concurrently, Axovant's majority shareholder, Roivant Sciences GmbH (Basel, Switzerland), purchased $25 million in Axovant shares at $1.75 in a private placement. Axovant had $154.3 million in cash at March 31.
Also on Wednesday, Axovant named Fraser Wright chief technology officer. He was co-founder and CTO at Spark Therapeutics Inc. (NASDAQ:ONCE), where he oversaw process development and manufacturing of Luxturna voretigene neparvovec-rzyl, the first FDA-approved in vivo gene therapy. Luxturna is an adeno-associated virus (AAV) delivering the retinal pigment epithelium-specific protein 65kDa (RPE65) gene. It is approved to treat biallelic RPE65 mutation-associated retinal dystrophy.
Axovant restructured this year after the departures of top executives and board members, including director and CEO David Hung. The company's current CEO is Pavan Cheruvu.
In Wednesday's deal, Axovant also obtained rights from Oxford BioMedica to AXO-Lenti-PD's predecessor therapy, ProSavin. Data published in The Lancet in 2014 showed the therapy significantly improved mean Unified Parkinson's Disease Rating Scale (UPDRS) part III off medication scores from baseline at six months, meeting a co-primary endpoint.
Both AXO-Lenti-PD and ProSavin deliver genes encoding tyrosine hydroxylase (TYH), dopa decarboxylase (DDC; AADC) and a cofactor. Axovant said the newer therapy delivers the genes with a modified payload configuration.
Oxford BioMedica was up 136p (19%) to 860p in intraday trading on Wednesday.